Nothing Special   »   [go: up one dir, main page]

AU728220B2 - Methods for increasing the efficiency of recombinant AAV product - Google Patents

Methods for increasing the efficiency of recombinant AAV product Download PDF

Info

Publication number
AU728220B2
AU728220B2 AU69741/98A AU6974198A AU728220B2 AU 728220 B2 AU728220 B2 AU 728220B2 AU 69741/98 A AU69741/98 A AU 69741/98A AU 6974198 A AU6974198 A AU 6974198A AU 728220 B2 AU728220 B2 AU 728220B2
Authority
AU
Australia
Prior art keywords
aav
rep
raav
expression
gene
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired
Application number
AU69741/98A
Other languages
English (en)
Other versions
AU6974198A (en
Inventor
Richard J Samulski
Richard Snyder
Xiao Xiao
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of North Carolina at Chapel Hill
Cell Genesys Inc
Original Assignee
University of North Carolina at Chapel Hill
Cell Genesys Inc
University of North Carolina System
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of North Carolina at Chapel Hill, Cell Genesys Inc, University of North Carolina System filed Critical University of North Carolina at Chapel Hill
Publication of AU6974198A publication Critical patent/AU6974198A/en
Application granted granted Critical
Publication of AU728220B2 publication Critical patent/AU728220B2/en
Anticipated expiration legal-status Critical
Expired legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU69741/98A 1997-04-14 1998-04-14 Methods for increasing the efficiency of recombinant AAV product Expired AU728220B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4354797P 1997-04-14 1997-04-14
US60/043547 1997-04-14
PCT/US1998/007654 WO1998046728A1 (en) 1997-04-14 1998-04-14 Methods for increasing the efficiency of recombinant aav product

Publications (2)

Publication Number Publication Date
AU6974198A AU6974198A (en) 1998-11-11
AU728220B2 true AU728220B2 (en) 2001-01-04

Family

ID=21927717

Family Applications (1)

Application Number Title Priority Date Filing Date
AU69741/98A Expired AU728220B2 (en) 1997-04-14 1998-04-14 Methods for increasing the efficiency of recombinant AAV product

Country Status (10)

Country Link
US (2) US6548286B1 (ja)
EP (1) EP1007637B1 (ja)
JP (2) JP4135120B2 (ja)
AT (1) ATE270324T1 (ja)
AU (1) AU728220B2 (ja)
CA (1) CA2287478C (ja)
DE (1) DE69824859T2 (ja)
ES (1) ES2224375T3 (ja)
NZ (1) NZ500546A (ja)
WO (1) WO1998046728A1 (ja)

Families Citing this family (133)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4135120B2 (ja) * 1997-04-14 2008-08-20 セル ジェネシス，インコーポレーテッド 組換えａａｖ生産の効率を上昇させる方法
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
JP2003511037A (ja) * 1999-10-01 2003-03-25 ジェノボ， インコーポレイテッド ＡＡＶｒｅｐ／ｃａｐ遺伝子を含むアデノウイルスを使用する組換えＡＡＶの産生
US7115391B1 (en) 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
JP2002153278A (ja) * 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
US20040197312A1 (en) 2003-04-02 2004-10-07 Marina Moskalenko Cytokine-expressing cellular vaccine combinations
US7485291B2 (en) * 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
CA2526120A1 (en) * 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
US20050136035A1 (en) * 2003-06-03 2005-06-23 Derek Ko Cell specific replication-competent viral vectors comprising a self processing peptide cleavage site
US7186699B2 (en) * 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
US7700095B2 (en) 2004-03-19 2010-04-20 Yale University Detection, isolation and uses of renalase (monoamine oxidase c)
AU2005287393A1 (en) * 2004-06-18 2006-03-30 The University Of Montana AAV mediated gene delivery to cochlear cells
EP1765846A4 (en) * 2004-07-13 2010-02-17 Cell Genesys Inc AAV VECTOR COMPOSITIONS AND ITS USE IN PROCESSES FOR INCREASING IMMUNOGLOBULIN EXPRESSION
US20060293264A1 (en) * 2004-07-22 2006-12-28 Grandis Jennifer R STAT3 decoy oligonucleotides and uses therefor
EP2484780A1 (en) 2004-07-23 2012-08-08 The University of North Carolina At Chapel Hill Methods and materials for determining pain sensibility and predicting and treating related disorders
US20060057127A1 (en) * 2004-09-10 2006-03-16 Pocheng Liu Cytokine-expressing cellular vaccines for treatment of prostate cancer
US20060234347A1 (en) * 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
US7632509B2 (en) * 2005-07-19 2009-12-15 Biosante Pharmaceuticals, Inc. Methods to express recombinant proteins from lentiviral vectors
EP2484774A3 (en) 2005-07-21 2012-11-14 Abbott Laboratories Multiple gene expression including sorf contructs and methods with polyproteins, pro-proteins, and proteolysis
EP1981548A4 (en) * 2006-01-20 2010-03-24 Univ North Carolina ENHANCED PRODUCTION OF INFECTIOUS VECTORS OF PARVOVIRUS IN INSECT CELLS
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
EP2013348A4 (en) 2006-03-30 2009-09-02 Univ California METHODS AND COMPOSITIONS FOR LOCALIZED SECRETION OF ANTI-CTLA-4 ANTIBODIES
US7919079B2 (en) * 2006-03-31 2011-04-05 Biosante Pharmaceuticals, Inc. Cancer immunotherapy compositions and methods of use
US20070292922A1 (en) * 2006-03-31 2007-12-20 Cell Genesys, Inc. Regulated expression of recombinant proteins from adeno-associated viral vectors
EP2848253A1 (en) 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor VIII and factor IX genes and vectors for gene therapy
US20090028857A1 (en) 2007-07-23 2009-01-29 Cell Genesys, Inc. Pd-1 antibodies in combination with a cytokine-secreting cell and methods of use thereof
WO2009032256A2 (en) 2007-08-30 2009-03-12 Cell Genesys, Inc. Apc activators in combination with a cytokine-secreting cell and methods of use thereof
WO2009105135A1 (en) 2008-02-19 2009-08-27 Celladon Corporation Compositions for enhanced uptake of viral vectors in the myocardium
US8840881B2 (en) 2008-08-28 2014-09-23 Aduro Gvax Inc. Methods and compositions for treating prostate cancer or inducing a humoral immune response against prostate cancer
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
CN102812040A (zh) 2009-10-30 2012-12-05 雅培制药有限公司 Sorf构建体和多基因表达
WO2011084694A1 (en) 2009-12-17 2011-07-14 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Stabilized stat3 decoy oligonucleotides and uses therefor
BR112012024934A2 (pt) 2010-03-29 2016-12-06 Univ Pennsylvania sistemas de ablação de transgene induzida farmacologicamente
EP2605798A1 (en) 2010-07-12 2013-06-26 Universidad Autònoma de Barcelona Gene therapy composition for use in diabetes treatment
EP2809347A1 (en) 2012-02-01 2014-12-10 The Arizona Board Of Regents On Behalf Of The University Of Arizona Campylobacter immunogenic compositions and uses thereof
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
DE102013004595A1 (de) 2013-03-15 2014-09-18 Emergent Product Development Germany Gmbh RSV-Impfstoffe
WO2014201252A2 (en) * 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
CA2924013A1 (en) 2013-11-05 2015-05-14 Clontech Laboratories, Inc. Dry transfection compositions and methods for making and using the same
GB201322626D0 (en) 2013-12-19 2014-02-05 Immutep S A Combined preparations for the treatment of cancer
WO2015138348A1 (en) 2014-03-09 2015-09-17 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
JP6983511B2 (ja) 2014-04-25 2021-12-17 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 脳中の転移乳癌および他の癌を処置するための方法および組成物
SG11201609207SA (en) 2014-05-13 2016-12-29 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
CA2974235A1 (en) 2015-01-07 2016-07-14 Universitat Autonoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
GB201500374D0 (en) 2015-01-09 2015-02-25 Immutep S A Combined preparations for the treatment of cancer
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
SG11201707063TA (en) 2015-03-02 2017-09-28 Adverum Biotechnologies Inc Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
EP3288594B1 (en) 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
WO2016200543A2 (en) 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
EP3325018A4 (en) 2015-07-22 2019-04-24 Duke University HIGH EFFICIENCY SCREENING OF REGULATORY ELEMENT FUNCTION USING EPIGENOUS EDITING TECHNOLOGIES
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
CA2995733A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
IL296929A (en) 2015-09-24 2022-12-01 Univ Pennsylvania A preparation and method for the treatment of a complement-mediated disease
ES2907486T3 (es) 2015-09-24 2022-04-25 Univ North Carolina Chapel Hill Métodos y composiciones para reducir metástasis
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
US11970710B2 (en) 2015-10-13 2024-04-30 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
JP2019501115A (ja) 2015-10-28 2019-01-17 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝子治療のためのアデノ随伴ウイルスベクターのくも膜下腔内投与
EP3400304B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JP7057281B2 (ja) 2015-12-14 2022-04-19 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患のための遺伝子療法
US11241506B2 (en) 2015-12-14 2022-02-08 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
CA3022600A1 (en) 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
CA3029646A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
ES2982489T3 (es) 2016-08-19 2024-10-16 Univ Florida Composiciones para el tratamiento de afecciones que utilizan virus adeno-asociados recombinantes autocomplementarios
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US20190328846A1 (en) 2016-12-01 2019-10-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Pharmaceutical compositions for the treatment of retinal degenerative diseases
JP7282378B2 (ja) 2016-12-07 2023-05-29 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド ＩＬ－１Ｒａ ｃＤＮＡ
KR20190118163A (ko) 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SI3589730T1 (sl) 2017-02-28 2024-04-30 The Trustees Of The University Of Pennsylvania Adeno-povezan virus (AAV) clade F vektor in njihova uporaba
KR20190135000A (ko) 2017-02-28 2019-12-05 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Aav 벡터를 기반으로 하는 인플루엔자 백신
JP7211960B2 (ja) 2017-03-01 2023-01-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患の遺伝子治療
EP3596213A4 (en) 2017-03-17 2021-02-17 Adverum Biotechnologies, Inc. COMPOSITIONS AND PROCEDURES FOR ENHANCED GENE EXPRESSION
GB201706090D0 (en) * 2017-04-18 2017-05-31 Glaxosmithkline Ip Dev Ltd Methods for adeno-associated viral vector production
CN110785495A (zh) 2017-04-18 2020-02-11 葛兰素史克知识产权开发有限公司 腺相关病毒载体的生产方法
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
RU2019139555A (ru) 2017-05-11 2021-06-11 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания Генная терапия нейрональных цероидных липофусцинозов
CN110913886A (zh) 2017-05-24 2020-03-24 巴塞罗那自治大学 包含成纤维细胞生长因子21(fgf21)编码序列的病毒表达构建体
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EP3954766A1 (en) 2017-10-10 2022-02-16 NantBio, Inc. Modified ec7 cells having low toxicity to viral production payloads
AU2018392480A1 (en) 2017-12-19 2020-07-02 Akouos, Inc. AAV-mediated delivery of therapeutic antibodies to the inner ear
GB201800903D0 (en) 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
US20230040603A1 (en) 2018-10-01 2023-02-09 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
CA3120923A1 (en) 2018-11-26 2020-06-04 Universitat Autonoma De Barcelona Fibroblast growth factor 21 (fgf21) gene therapy
BR112021015817A2 (pt) 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn
CA3130055A1 (en) 2019-02-26 2020-09-03 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
WO2020239995A1 (en) 2019-05-31 2020-12-03 Universitat Autònoma De Barcelona Insulin gene therapy
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
BR112022011332A2 (pt) 2019-12-10 2022-08-23 Takeda Pharmaceuticals Co Vetor de vírus adeno-associado recombinante, vírus adeno-associado recombinante, e, método para tratar um indivíduo com síndrome de hunter
TW202208405A (zh) 2020-05-13 2022-03-01 美商阿科奧斯公司 用於治療ｓｌｃ２６ａ４相關聽力損失之組合物及方法
AU2021281506A1 (en) 2020-05-26 2023-02-02 Universitat Autònoma De Barcelona Fibroblast growth factor 21 (FGF21) gene therapy for central nervous system disorders
CN116438312A (zh) 2020-08-24 2023-07-14 宾夕法尼亚州大学信托人 编码glp-1受体激动剂融合物的病毒载体和其在治疗代谢性疾病中的用途
KR20230058102A (ko) 2020-08-26 2023-05-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn 관련 성인 발병 신경변성 치료를 위한 재조합 아데노-연관 바이러스
KR20230083287A (ko) 2020-10-07 2023-06-09 리젠엑스바이오 인크. Cln2 질환의 안구 징후에 대한 유전자 요법
GB202016254D0 (en) * 2020-10-13 2020-11-25 Freeline Therapeutics Ltd Plasmid system
AR123838A1 (es) 2020-10-18 2023-01-18 Univ Pennsylvania Vector mejorado de virus adenoasociado (aav) y usos de este
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
US20230407333A1 (en) 2020-10-29 2023-12-21 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
JP2023551533A (ja) 2020-12-01 2023-12-08 アコーオス インコーポレイテッド 前庭神経鞘腫関連症状を治療するための抗ｖｅｇｆ抗体構築物及び関連する方法
CN116670152A (zh) 2020-12-01 2023-08-29 宾夕法尼亚州大学信托人 具有组织特异性靶向基序的新型组合物和含有其的组合物
KR20230127263A (ko) 2020-12-29 2023-08-31 아카우오스, 인크. Clrn1-연관된 청력손실 및/또는 시력손실을 치료하기위한 조성물 및 방법
WO2022162067A1 (en) 2021-01-30 2022-08-04 Universitat Autònoma De Barcelona Gene therapy for monogenic diabetes
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AU2022227017A1 (en) 2021-02-26 2023-09-07 Takeda Pharmaceutical Company Limited Composition and methods for the treatment of fabry disease
EP4323520A1 (en) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
CA3216146A1 (en) 2021-04-23 2022-10-27 Douglas Anderson Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
EP4089171A1 (en) 2021-05-12 2022-11-16 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Recombinant tert-encoding viral genomes and vectors
CN118475612A (zh) 2021-08-31 2024-08-09 斯科特生物公司 抗原结合分子及其用途
EP4408872A1 (en) 2021-09-30 2024-08-07 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
IL314434A (en) 2022-01-25 2024-09-01 Univ Pennsylvania AAV capsids for enhanced cardiac transduction and liver targeting neutralization
AR128422A1 (es) 2022-02-02 2024-05-08 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
WO2023187728A1 (en) 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024042485A1 (en) 2022-08-25 2024-02-29 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024052413A1 (en) 2022-09-07 2024-03-14 Universitat Autònoma De Barcelona Beta-hexosaminidase vectors
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
JP3952312B2 (ja) * 1993-11-09 2007-08-01 メディカル カレッジ オブ オハイオ アデノ関連ウイルス複製遺伝子を発現可能な安定な細胞株
US5693531A (en) * 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6027931A (en) 1995-08-03 2000-02-22 Avigen, Inc. High-efficiency AA V helper functions
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
JP4135120B2 (ja) * 1997-04-14 2008-08-20 セル ジェネシス，インコーポレーテッド 組換えａａｖ生産の効率を上昇させる方法
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production

Also Published As

Publication number Publication date
DE69824859D1 (de) 2004-08-05
EP1007637A1 (en) 2000-06-14
AU6974198A (en) 1998-11-11
US7229823B2 (en) 2007-06-12
JP4249239B2 (ja) 2009-04-02
WO1998046728A1 (en) 1998-10-22
JP2001520516A (ja) 2001-10-30
ATE270324T1 (de) 2004-07-15
US20030211614A1 (en) 2003-11-13
JP2008043349A (ja) 2008-02-28
DE69824859T2 (de) 2005-08-04
EP1007637A4 (en) 2002-04-03
US6548286B1 (en) 2003-04-15
NZ500546A (en) 2001-09-28
CA2287478A1 (en) 1998-10-22
JP4135120B2 (ja) 2008-08-20
CA2287478C (en) 2007-06-19
ES2224375T3 (es) 2005-03-01
EP1007637B1 (en) 2004-06-30

Similar Documents

Publication Publication Date Title
AU728220B2 (en) Methods for increasing the efficiency of recombinant AAV product
US20190100773A1 (en) Metabolically activated recombinant viral vectors and methods for their preparation and use
US6037177A (en) Method for increasing the efficiency of recombinant AAV production
EP0844887A1 (en) Aav transduction of myoblasts
Xiao et al. Adeno-associated virus (AAV) vectors for gene transfer
EP1916258B1 (en) Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
AU2005202946B2 (en) Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
AU2008207383A1 (en) Enhancement of expression of a single-stranded heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs